CD72 Nano-Cars for the Treatment of MLLr B-Cell Acute Lymphoblastic Leukemia
Abstract
The goal of this proposal is to preclinically develop a new therapeutic option for pediatric and young adult (PAYA) individuals with a poor prognosis hematologic malignancy: B-cell acute lymphoblastic leukemia carrying a translocation of the KMT2A/MLL gene. Specifically, this work is motivated by our recent discovery that CD72 is a surface antigen specifically upregulated on this poor-prognosis tumor subtype, and the further discovery of initial efficacious, proof-of-principle CAR-T cells against this target (Nix et al, Cancer Discov 2021). The Specific Aims of this award are to 1) further optimize a leading clinical CAR-T design as a true therapeutic candidate; 2) perform process development studies required to manufacture a GMP-like CAR-T cell and obtain other data required for a successful pre-IND meeting with the FDA; 3) develop a clinical trial plan and assemble and complete the full pre-IND submission. In Year 1 of this award, we have accomplished our goals described in Aim 1 by establishing a true therapeutic candidate CAR-T design and obtaining a GMP-like lentiviral construct from the City of Hope manufacturing facility, as planned. In accordance with our timeline, and are now prepared to fully engage in process engineering, efficacy, and pharmacology/toxicology/biodistribution studies in Year 2.
Document Details
- Document Type
- Technical Report
- Publication Date
- Aug 01, 2023
- Accession Number
- AD1216083
Entities
People
- Adila Izgutdina
- Akul Naik
- Arun Wiita
- Szu-ying (sharon) Chen
- William Temple
Organizations
- University of California Regents